Teaching tolerance: New approaches to enzyme replacement therapy for Pompe disease

New paper on Tregitopes Published: DOI: https://dx.doi.org/10.4161/hv.21405

Authored by Leslie Cousens, Federico Mignozzi, Sander van der Marel, Yan Su, Richard Garman, Valerie Ferreira, William Martin, David W. Scott and Anne S. De Groot

Babies born with Pompe disease require life-long treatment with enzyme-replacement therapy (ERT). Despite the human origin of the therapy, recombinant human lysosomal acid α glucosidase (GAA, rhGAA), ERT unfortunately leads to the development of high titers of anti-rhGAA antibody, decreased effectiveness of ERT, and a fatal outcome for a significant number of children who have Pompe disease. The severity of disease, anti-drug antibody (ADA) development, and the consequences thereof are directly related to the degree of the enzyme deficiency….continue reading